Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12617000690358
Ethics application status
Approved
Date submitted
11/05/2017
Date registered
15/05/2017
Date last updated
3/05/2018
Type of registration
Prospectively registered

Titles & IDs
Public title
The Duchenne Registry Australia
Scientific title
The Duchenne Registry Australia - a patient registry to collect information about Australians with Duchenne or Becker muscular dystrophy and female carriers of these conditions
Secondary ID [1] 291917 0
Nil known
Universal Trial Number (UTN)
U1111-1196-5736
Trial acronym
TDRA
Linked study record
NCT02069756

Health condition
Health condition(s) or problem(s) studied:
Duchenne muscular dystrophy 303237 0
Becker muscular dystrophy 303238 0
manifesting carriers of Duchenne 303239 0
carriers of Duchenne or Becker muscular dystrophy (asymptomatic) 303240 0
Condition category
Condition code
Musculoskeletal 302664 302664 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Observational
Patient registry
True
Target follow-up duration
40
Target follow-up type
Years
Description of intervention(s) / exposure
The Duchenne Registry Australia is an online patient/caregiver self-report registry collecting data about Australians with Duchenne and Becker muscular dystrophy. Longitudinal data including demographics, disease manifestations, healthcare utilisation, and quality of life of individuals with Duchenne and Becker muscular dystrophy (DBMD) and carriers of these conditions are collected, curated, and maintained in accordance with Australian Privacy Laws and Guidelines.
Participants explicitly permit the collection and use of their data through an online consent or parental permission process and form. Patients’ anonymised data are used to facilitate research, speed the development of and recruitment for clinical trials, and generate potential research hypotheses. In addition, registrants are informed about clinical trials, clinical research, and social/behavioural research studies that may be of interest; they then choose whether to seek additional information and/or to participate.
Intervention code [1] 298039 0
Not applicable
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 302068 0
Genetic mutation
Timepoint [1] 302068 0
Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime. Genetic mutation data is collected by patient report and verified by curation/review of genetic test report when provided.
Secondary outcome [1] 334676 0
Ambulation status. Ambulation status is assessed from several questions about mobility in home and away from home, ability to sit and stand, use of assistive devices, and age at full time wheelchair use.
Timepoint [1] 334676 0
Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime.
Secondary outcome [2] 334677 0
Corticosteroid use. Data collected includes whether or not corticosteroids are used, and if so, name of corticosteroid, age started/stopped, dose and dosing regimen.
Timepoint [2] 334677 0
Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime.
Secondary outcome [3] 334678 0
Cardiovascular status. Multiple questions regarding cardiovascular symptoms, age at diagnosis of cardiomyopathy, use of cardiac medications, date of most recent echocardiogram and/or cardiac MRI, and the LVEF and/or LVSF value.
Timepoint [3] 334678 0
Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime.
Secondary outcome [4] 334679 0
Respiratory status. Assessed from questions about use of breathing devices, age at which breathing devices were started, date of most recent pulmonary function test (spirometry), and the FVC value (% predicted and raw FVC in liters).
Timepoint [4] 334679 0
Registrants are requested to update their medical history every 6-12 months, and they will be followed throughout their lifetime.

Eligibility
Key inclusion criteria
Diagnosis of Duchenne or Becker muscular dystrophy; Manifesting female carriers and asymptomatic female carriers also included in registry.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Diagnosis of any other type of muscular dystrophy (including limb-girdle muscular dystrophy).

Study design
Purpose
Natural history
Duration
Longitudinal
Selection
Convenience sample
Timing
Both
Statistical methods / analysis
There are no accurate figures on the number of individuals in Australia with DBMD, but we estimate that there is between 600 and 900 and we will encourage all of them to register. Female carriers will also be encouraged to register. Registrants will be requested to update their accounts at least annually and will continue to enrol participants indefinitely, to facilitate better characterisation of DBMD and provide individuals with the benefits of registration.

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
1/06/2017
Actual
1/06/2017
Date of last participant enrolment
Anticipated
1/06/2027
Actual
Date of last data collection
Anticipated
1/06/2047
Actual
Sample size
Target
1000
Accrual to date
120
Final
Recruitment in Australia
Recruitment state(s)
ACT,NSW,NT,QLD,SA,TAS,WA,VIC

Funding & Sponsors
Funding source category [1] 296423 0
Charities/Societies/Foundations
Name [1] 296423 0
Save Our Sons Duchenne Foundation
Country [1] 296423 0
Australia
Primary sponsor type
Charities/Societies/Foundations
Name
Save Our Sons Duchenne Foundation
Address
PO Box 350, Marrickville
NSW 2204
Country
Australia
Secondary sponsor category [1] 295375 0
None
Name [1] 295375 0
Address [1] 295375 0
Country [1] 295375 0
Other collaborator category [1] 279567 0
Charities/Societies/Foundations
Name [1] 279567 0
Parent Project Muscular Dystrophy/DuchenneConnect
Address [1] 279567 0
401 Hackensack Ave, Hackensack, NJ 07601
Country [1] 279567 0
United States of America

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 297650 0
Bellberry Limited
Ethics committee address [1] 297650 0
129 Glen Osmond Road Eastwood South Australia 5063
Ethics committee country [1] 297650 0
Australia
Date submitted for ethics approval [1] 297650 0
31/01/2017
Approval date [1] 297650 0
14/03/2017
Ethics approval number [1] 297650 0
2016-11-838

Summary
Brief summary
The Duchenne Registry Australia is an online, patient-report registry for individuals with Duchenne and Becker muscular dystrophy and carrier females. The purpose of The Duchenne Registry Australia is to connect Duchenne and Becker patients with actively recruiting clinical trials and research studies, and to educate patients and families about Duchenne and Becker care and research. At the same time, the registry is a valuable resource for clinicians and researchers in academia and industry, allowing access to a de-identified, aggregate dataset provided by patients and their families—information that is vital to advances in the care and treatment of Duchenne.
Trial website
www.duchenneregistry.org.au
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 74706 0
Dr Kristina Elvidge
Address 74706 0
Research Affairs Manager - Save Our Sons Duchenne Foundation and
DuchenneConnect Australia Registry Coordinator
PO Box 230
Coburg, VIC, 3058
Country 74706 0
Australia
Phone 74706 0
(+61) 1800 940 492
Fax 74706 0
Email 74706 0
[email protected]
Contact person for public queries
Name 74707 0
Dr Kristina Elvidge
Address 74707 0
Research Affairs Manager - Save Our Sons Duchenne Foundation and
DuchenneConnect Australia Registry Coordinator
PO Box 230
Coburg, VIC, 3058
Country 74707 0
Australia
Phone 74707 0
(+61) 1800 940 492
Fax 74707 0
Email 74707 0
[email protected]
Contact person for scientific queries
Name 74708 0
Dr Kristina Elvidge
Address 74708 0
Research Affairs Manager - Save Our Sons Duchenne Foundation and
DuchenneConnect Australia Registry Coordinator
PO Box 230
Coburg, VIC, 3058
Country 74708 0
Australia
Phone 74708 0
(+61) 1800 940 492
Fax 74708 0
Email 74708 0
[email protected]

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
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